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In a Dec. 2, 2014, webcast for the Duchenne MD community, Sarepta Therapeutics explains plans, answers questions; the presentation is available on Sarepta's site

posted on December 2, 2014 - 2:11pm

Cambridge, Mass.-based Sarepta Therapeutics, developer of eteplirsen and other "exon-skipping" drugs designed to treat Duchenne muscular dystrophy (DMD), provided overall reassurance about eteplirsen's development and some details of future plans to the DMD community in a Dec.

To people faced with life-threatening diseases, the U.S. Food and Drug Administration (FDA) can seem like an indifferent obstacle, keeping them from treatments that would otherwise be available. But the reality is much more layered and complex.
Here, MDA answers some frequently asked questions about how the FDA works to shed light on this topic.
Q: Why does the FDA have stringent...

Abby Bronson is the Duchenne muscular dystrophy program manager at Children's National Medical Center in Washington, D.C., where she works with various stakeholders to further therapeutic development for this disease. Bronson has a master's degree in business administration from the University of Pennsylvania and has managed new product development and marketing at biotechnology and...

Arthur Caplan is the William F. and Virginia Connolly Mitty Professor and founding head of the Division of Bioethics at New York University Langone Medical Center in New York City. He holds a doctoral degree in the history and philosophy of science from Columbia University, has authored or edited 32 books and more than 600 papers, and has served on national and international committees as an...

Richard Klein is the director of the Patient Liaison Program at the Office of Health and Constituent Affairs at the U.S. Food and Drug Administration (FDA).
Q: What is the most common misconception that patient communities have about the FDA's accelerated approval mechanism?

Neuromuscular disease specialist and biotechnology company employee Timothy Miller says it can be a significant risk and financial hardship for a small company to supply a drug that has not been through the full approval process

Timothy Miller has been a university-associated neurologist specializing in neuromuscular disorders who has recently moved to the biotechnology industry. He has been the director of the MDA-supported pediatric neuromuscular disorders clinic at Children's Clinics for Rehabilitative Services in Tucson, Ariz.; an assistant professor of neurology, pathology and pediatrics at the University of Arizona...

Update (April 15, 2014): This story has been updated to reflect that there is now a new FDA office, the Office of Health and Constituent Affairs, whose job it is to serve as a liaison between the FDA and various outside stakeholders, including patient advocates and consumers. See Resources, below.
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